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February 14, 2017

When Will Mendelian Randomization Become Relevant for Clinical Practice and Public Health?

Author Affiliations
  • 1MRC Integrative Epidemiology Unit (IEU) at the University of Bristol, Bristol, United Kingdom
JAMA. 2017;317(6):589-591. doi:10.1001/jama.2016.21189

Obtaining reliable evidence regarding factors that cause disease or influence disease progression is clearly central to progress in science. There have been many high-profile failures in this regard, which range from health-related behaviors (eg, dietary components that observational epidemiology strongly suggested protected against chronic disease that failed when tested in large-scale randomized clinical trials [RCTs]), through vitamin supplement use and menopausal hormone therapy, to a large number of drugs that failed at phase 3 trial stage.1,2 Observational epidemiologic studies are prone to confounding, reverse causation (ie, when the disease process influences the exposure, rather than vice versa), and a variety of other biases.1 Furthermore, laboratory studies at the cellular through to the whole-animal level have also led to many erroneous conclusions being drawn.3

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